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Genentech Aims to Set Precedent With FDA OK in Early Breast Cancer

Exactly when does it make sense to start giving breast cancer patients one of the most potent, targeted drugs that doctors have in their repertoire?

That’s the gist of a question that an FDA panel of cancer drug experts will consider on Thursday, when they review an application from South San Francisco-based Genentech, a unit of Switzerland-based Roche. On the surface, the company is simply asking the FDA for clearance to start selling one of its targeted antibody drugs to women with locally confined breast cancer that overexpresses the HER2 protein, and who haven’t yet had surgery to remove breast tumors. This would be a first, as there currently are no FDA-approved drugs in this group of women, who are sometimes called “neoadjuvant” or “preoperative” patients.

The question might sound like a narrow one of medical interest, but it has bigger implications for Genentech and the biotech industry. For Genentech, a yes from the FDA would obviously open the door to a larger potential market for pertuzumab (Perjeta), a targeted drug that won FDA approval last year for a sicker group of patients who have breast cancer that has spread.

But this application is really about more than just one drug in one big company’s portfolio. If Genentech gets its way, it could change the way the company and others think about developing cancer drugs, enabling them to consider clinical trials that are faster, cheaper, and smaller. Put another way, the FDA will be signaling that, in certain circumstances, it is willing to approve new cancer drugs without forcing companies to make huge investments in time and money to gather proof from large studies that they keep tumors in check for a few extra months, or ultimately help patients live longer.

“We are setting a precedent,” says Dietmar Berger, Genentech’s vice president of clinical oncology. “We very much welcome that FDA is using this type of innovation. What we’re trying to do is bring a drug to patients, but to get the right amount of efficacy and safety information that you need.”

Essentially, the FDA panel on Thursday will be a referendum of sorts on how much faith the agency is willing to put in a leading indicator of patient prognosis called pathological complete response (pCR). In the case of this Genentech drug, the company gathered that data, on the percentage of patients who appeared free of cancer, between nine and 18 weeks after they entered a clinical trial. While that kind of result should logically give a patient a better future, no one can say for sure if that early positive sign translates into a long-term improvement in survival time and quality of life.

If the FDA says that the pCR measurement is valid, you can be sure many other companies will try to design trials to hit that goal for other kinds of cancer. Trials of this nature could … Next Page »